Compassionate Drug Use

What is compassionate drug use?

Medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available.

Drugs that are being tested but have not yet been approved by the US Food and Drug Administration (FDA) are called investigational drugs. These drugs are generally available only to people who are taking part in a clinical trial (a research study that is testing the drug).

Being able to use one of these drugs when you are not in a clinical trial is most commonly referred to as compassionate use.

Is compassionate drug use legal?

Compassionate drug use is legal, but it’s tightly restricted to people who meet certain conditions. For people who aren’t in clinical trials, a drug company can allow use of their unapproved drug in 2 ways:

Through expanded access programs

Through single patient access

Expanded access programs

A company sponsoring a drug in the late stages of drug development, such as Phase III clinical trials, can offer expanded access programs (EAPs) for patients who are not able to enroll in a clinical trial. (See Clinical Trials: What You Need to Know to learn more about the phases of clinical trials.)

The FDA generally approves these EAPs if the ongoing clinical trials have shown that the drug works at least somewhat to treat cancer. This can allow a lot of people access to the unapproved drug, as long as they meet the requirements of the EAP.

Single patient access

Patients who don’t qualify for either clinical trials or an expanded access program (if one exists) may be able to get the unapproved new drug by applying for single patient access.

In this case, the patient’s doctor must first ask the drug company if the drug can be used for the patient and see if the drug company will supply it. If the company agrees, the doctor works with the drug company to ask the FDA to approve the drug for use by this one patient.

The FDA requires the doctor to send information about the patient, why the request is being made, the proposed treatment plan, and a signed informed consent from the patient. (See Informed Consent for more on this.) The length of time it takes to get single patient access varies. But if it’s an emergency, the FDA can complete the paperwork in 24 hours.

Who might benefit from using unapproved drugs?

According to guidelines from the National Cancer Institute, most compassionate drug use is for patients who meet all of these conditions:

Have used standard treatments and they have not worked

Are not eligible for any current clinical trial that’s using the drug

Have no other treatment options

Have a type of cancer for which there’s reason to expect the investigational drug will help

Are likely to have benefits that outweigh the risks involved

Is it difficult to get drugs for compassionate use?

Perhaps the biggest problem with compassionate drug use is that it’s often hard to get the drug. The simplest way to get an unapproved drug is through a clinical trial. But many people with life-threatening diseases can’t find suitable clinical trials, live far from cancer research centers, or are not eligible for any studies being done.

Getting the drug through expanded access programs (if one is offered by the drug company) or single-patient compassionate use is possible for some people. But going through all the steps needed to get single-patient compassionate use of an unapproved drug can be frustrating and take a lot of time. For instance, drug companies have different policies and processes. And there’s no way to force the drug company to supply the drug. There may be very limited amounts of the drug available for compassionate use, or drug companies may only have enough drug for use in clinical trials. (Producing extra medicine for people who are not in clinical trials can be costly for the drug company, especially when there’s a chance the drug might never be approved.)

Compassionate drug use can also be very confusing. There are several programs that regulate it. Many terms and definitions are used to describe how a patient may get access to an unapproved drug outside of a clinical trial. Drug companies, patient advocacy groups, and the FDA all may use different terms for the same things. The FDA provides definitions for the terms they use in their regulations. But most drug companies use different terms that are unique to their specific compassionate access programs.

Another big problem is cost. Some drug companies will supply the drug for free, but others charge patients. Most insurance companies will not pay for investigational drugs. There may also be other costs, such as the clinic’s cost of giving the drug and monitoring your response, that might not be covered by your health insurance.

Still, despite these hurdles, compassionate drug use does happen. Because actual use is not well-documented, there are no numbers or statistics on how often it’s done, who’s doing it, or how well it’s working for patients.

What should I ask my doctor about compassionate drug use?

Here are some questions you may want to ask if your doctor is thinking about compassionate drug use:

Are there any approved treatments that I haven’t tried?

Is there any evidence to support the use of this drug to treat my type of cancer?

What makes you think this drug could help me?

In what way do you think this drug is likely to work better than an approved drug?

What are the known risks and benefits of treatment with this drug?

Will the drug company give me the drug for free? If not, how is it to be paid for? What costs will I have to pay to get the drug? Will my insurance cover any costs?

What will I have to do to get access to this drug?

How long do you think it will take for me to get access to this drug?

Where do I start if I want to apply for compassionate drug use?

Your doctor or one of the office staff will work with you on this process. The US Food and Drug Administration, Center for Drug Evaluation and Research is the starting point.

The main FDA number is 1-888-463-6332 (toll free). Select the option to “request information or speak with an FDA representative about an FDA-regulated product or program.” You may have to continue through the menu to reach someone to ask about the Emergency Use Investigational New Drug Program for Oncology Drugs.

US Food and Drug Administration. Physician Request for an Individual Patient IND under Expanded Access for Non-emergency or Emergency Use. 03/15/2016. Accessed at www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm107434.htm on April 5, 2016.

National Cancer Institute. Access to Investigational Drugs. August 4, 2009. Accessed at www.cancer.gov/about-cancer/treatment/drugs/investigational-drug-access-fact-sheet on April 5, 2016.

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